REAL-WORLD EFFECTIVENESS OF EMICIZUMAB PROPHYLAXIS IN PATIENTS WITH HEMOPHILIA A: A FIRST NATIONWIDE REPORT FROM SOUTH KOREA
Author(s)
JAEHEE WON, BS, MS, Eunjung Choo, BS, MS, HyunYung Lee, MPH, Hankil Lee, BS, MS, RPh, PhD.
Ewha Womans University, College of Pharmacy, Seoul, Korea, Republic of.
Ewha Womans University, College of Pharmacy, Seoul, Korea, Republic of.
OBJECTIVES: To characterize patients receiving emicizumab prophylaxis in South Korea and evaluate its real-world effectiveness.
METHODS: This retrospective observational cohort study included all patients receiving emicizumab prophylaxis in South Korea between May 1, 2020, and June 30, 2024. Baseline characteristics included sex, age, CCI score, and the number of patients with arthropathy. Effectiveness outcomes were stratified by inhibitor status: for patients with inhibitors (PWI), changes in zero-bleed rates and bypassing agent (BPA) prescription claims were compared between the 6-month periods pre- and post-emicizumab initiation; for patients without inhibitors (PWNI), changes in hospitalizations and emergency department (ED) visits were compared, while zero-bleed rates and FVIII prescription claims among patients with ≥1 such claim during the 6 months following initiation were assessed.
RESULTS: A total of 174 patients were included (PWI, n=28; PWNI, n=146). The median age (range) was 16.5 years (2.5-40.0) in PWI and 23.5 years (8.0-42.0) in PWNI, and all patients were male. Comparing the 6-month periods pre- and post-emicizumab initiation, the number of PWI achieving zero bleeds increased 2.3-fold (7→16), and mean BPA prescription claims (±SD) decreased by 89.0% (14.6±13.8→1.6±3.1). In PWNI, the number of hospitalized patients and those with ED visits decreased by 36.8% (19→12) and 59.1% (22→9), respectively. During the 6 months post-initiation, 120 PWNI (82.2%) achieved zero bleeds, and among patients with ≥1 FVIII claim, the mean claims (±SD) was 2.8±6.3.
CONCLUSIONS: Emicizumab prophylaxis demonstrated real-world effectiveness consistent with findings from RCTs, with substantial reductions in bleeding and healthcare utilization in both PWI and PWNI. As a first real-world analysis of emicizumab use in South Korea, these findings provide valuable early evidence to support its clinical use. Future studies with longer follow-up are warranted to further evaluate the long-term effectiveness and safety of emicizumab in routine clinical practice.
METHODS: This retrospective observational cohort study included all patients receiving emicizumab prophylaxis in South Korea between May 1, 2020, and June 30, 2024. Baseline characteristics included sex, age, CCI score, and the number of patients with arthropathy. Effectiveness outcomes were stratified by inhibitor status: for patients with inhibitors (PWI), changes in zero-bleed rates and bypassing agent (BPA) prescription claims were compared between the 6-month periods pre- and post-emicizumab initiation; for patients without inhibitors (PWNI), changes in hospitalizations and emergency department (ED) visits were compared, while zero-bleed rates and FVIII prescription claims among patients with ≥1 such claim during the 6 months following initiation were assessed.
RESULTS: A total of 174 patients were included (PWI, n=28; PWNI, n=146). The median age (range) was 16.5 years (2.5-40.0) in PWI and 23.5 years (8.0-42.0) in PWNI, and all patients were male. Comparing the 6-month periods pre- and post-emicizumab initiation, the number of PWI achieving zero bleeds increased 2.3-fold (7→16), and mean BPA prescription claims (±SD) decreased by 89.0% (14.6±13.8→1.6±3.1). In PWNI, the number of hospitalized patients and those with ED visits decreased by 36.8% (19→12) and 59.1% (22→9), respectively. During the 6 months post-initiation, 120 PWNI (82.2%) achieved zero bleeds, and among patients with ≥1 FVIII claim, the mean claims (±SD) was 2.8±6.3.
CONCLUSIONS: Emicizumab prophylaxis demonstrated real-world effectiveness consistent with findings from RCTs, with substantial reductions in bleeding and healthcare utilization in both PWI and PWNI. As a first real-world analysis of emicizumab use in South Korea, these findings provide valuable early evidence to support its clinical use. Future studies with longer follow-up are warranted to further evaluate the long-term effectiveness and safety of emicizumab in routine clinical practice.
Conference/Value in Health Info
2026-11, ISPOR Europe 2026, Vienna, Austria
Value in Health, Volume 29, Issue 12S
Code
RWD27
Topic
Clinical Outcomes, Epidemiology & Public Health, Real World Data & Information Systems
Topic Subcategory
Health & Insurance Records Systems
Disease
Alternative Medicine, Rare & Orphan Diseases, Systemic Disorders/Conditions (Anesthesia, Auto-Immune Disorders (n.e.c.), Hematological Disorders (non-oncologic), Pain)